Abstract
Purpose of review:
The goal of this review is to highlight some of the considerations involved in creating animal models to study rare bone diseases and then to compare and contrast approaches to creating such models, focusing on the advantages and novel opportunities offered by the CRISPR-Cas system.
Recent findings:
Gene editing after creation of double-stranded breaks in chromosomal DNA is increasingly being used to modify animal genomes. Multiple tools can be used to create such breaks, with the newest ones being based on the bacterial adaptive immune system known as CRISPR/Cas. Advances in gene editing have increased the ease and speed, while reducing the cost, of creating novel animal models of disease. Gene editing has also expanded the number of animal species in which genetic modification can be performed. These changes have significantly increased the options for investigators seeking to model rare bone diseases in animals.
Keywords:
Animal model; Gene-editing; Rare bone disease.
Publication types
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Research Support, N.I.H., Extramural
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Research Support, U.S. Gov't, Non-P.H.S.
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Review
MeSH terms
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Animals
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Bone Diseases / genetics*
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CRISPR-Cas Systems
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Calcium / deficiency
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Calcium, Dietary
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Collagen Type I / genetics
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Collagen Type I, alpha 1 Chain
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DNA Breaks, Double-Stranded
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Disease Models, Animal*
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Extracellular Matrix Proteins
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Gene Editing / methods*
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Gene Targeting / methods*
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Gene-Environment Interaction
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Low Density Lipoprotein Receptor-Related Protein-5 / genetics
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Mice
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Mice, Knockout
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Mice, Transgenic
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Molecular Chaperones
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Osteitis Deformans / genetics
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Osteitis Deformans / metabolism
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Osteogenesis Imperfecta / genetics
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Proteins / genetics
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Rabbits
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Rare Diseases / genetics*
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Rats
Substances
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Calcium, Dietary
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Col1a2 protein, mouse
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Collagen Type I
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Collagen Type I, alpha 1 Chain
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Crtap protein, mouse
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Extracellular Matrix Proteins
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Low Density Lipoprotein Receptor-Related Protein-5
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Lrp5 protein, mouse
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Molecular Chaperones
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Proteins
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Calcium
Supplementary concepts
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Osteoporosis-pseudoglioma syndrome